Amelioration of Leigh syndrome induced by mouse blastocyst complementation with a mutant human mitochondrial ATP synthase 6-Reference-Cited by-同舟云学术

Amelioration of Leigh syndrome induced by mouse blastocyst complementation with a mutant human mitochondrial ATP synthase 6

Published:2022-08-12 Issue:3 Volume:2 Page:
ISSN:2768-0622
Container-title:Clinical and Translational Discovery
language:en
Short-container-title:Clinical and Translational Dis

Author:

Yuan Huijun¹²^ORCID,Webster Keith A.³⁴,Bhatti Muhammad Tariq⁵,Hauswirth William W.⁶,Lewin Alfred S.⁷,Guy John¹

Affiliation:

1. Bascom Palmer Eye Institute University of Miami Miller School of Medicine Miami Florida USA

2. (Current) Division of Neonatology and Batchelor Children's Research Institute, Department of Pediatrics University of Miami Miller School of Medicine Miami Florida USA

3. Cullen Eye Institute, Department of Ophthalmology Baylor College of Medicine, Everglades Biopharma, LLC Houston Texas USA

4. Integene International, LLC Miami Florida USA

5. Department of Ophthalmology and Neurology Mayo Clinic College of Medicine Rochester Minnesota USA

6. Department of Ophthalmology University of Florida College of Medicine Gainesville Florida USA

7. Department of Molecular Genetics and Microbiology University of Florida College of Medicine Gainesville Florida USA

Funder

National Eye Institute

Research to Prevent Blindness

Publisher

Wiley

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ctd2.114

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4. Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome

5. Respiratory chain complex I deficiency: An underdiagnosed energy generation disorder